Titlebook: Antisense RNA Design, Delivery, and Analysis; Virginia Arechavala-Gomeza,Alejandro Garanto Book‘‘‘‘‘‘‘‘ 2022 The Editor(s) (if applicable)

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https://doi.org/10.1007/BFb0111518A development and other applications that have by and large utilized the same modifications. We also point out the pitfalls of the use of nucleic acids as drugs, such as their unwanted interactions with pattern recognition receptors, which can be mitigated by chemical modification or used as immunotherapeutic agents.

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Introduction and History of the Chemistry of Nucleic Acids TherapeuticsA development and other applications that have by and large utilized the same modifications. We also point out the pitfalls of the use of nucleic acids as drugs, such as their unwanted interactions with pattern recognition receptors, which can be mitigated by chemical modification or used as immunotherapeutic agents.

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Development and Use of Cellular Systems to Assess and Correct Splicing Defectsribe how to engineer splicing vectors, validate the reliability and reproducibility of alternative cellular systems, assess pre-mRNA splicing defects involved in IRD, and finally correct those by using antisense oligonucleotide-based strategies.